phase 3 clinical trial

Recombinant ADAMTS13 Therapy Demonstrates Superior Efficacy and Safety Over Plasma-Based Therapy in Congenital Thrombotic Thrombocytopenic Purpura: Final Phase 3 Trial Results

By MedXY 09/28/2026

Final phase 3 trial analysis confirms recombinant ADAMTS13 (rADAMTS13) provides effective prophylaxis with fewer acute and subacute TTP events and better safety than plasma-based therapy in congenital TTP patients.

Neurology news Pediatrics

Efficacy and Safety of Ecopipam for Tourette Syndrome: Phase 3 Randomized Clinical Trial

By MedXY 06/05/2026

This phase 3 trial found that ecopipam reduced tic relapse and maintained symptom improvement in children and adolescents with Tourette syndrome, with mainly central nervous system side effects and no major weight or metabolic concerns.

Oncology

Gedatolisib Demonstrates Promising Efficacy in Phase 3 VIKTORIA-1 Trial for HR+/HER2- Advanced Breast Cancer: NDA Submission Initiated

By MedXY 10/24/2025

Celcuity's phase 3 VIKTORIA-1 trial reveals that gedatolisib, a broad-spectrum PI3K/mTORC1/2 inhibitor, significantly improves progression-free survival in hormone receptor-positive, HER2-negative advanced breast cancer patients post CDK4/6 inhibitor therapy, prompting NDA rolling submission.

Clinical Updates Internal Medicine news Pediatrics Specialties

Oral Iptacopan Monotherapy in Paroxysmal Nocturnal Haemoglobinuria: 48-Week Outcomes from APPLY-PNH and APPOINT-PNH Phase 3 Trials

By MedXY 08/03/2025

Oral iptacopan provides sustained haemolysis control and improved haemoglobin in PNH patients over 48 weeks, with a favorable safety profile, supporting its role as a new therapeutic option.